Gene therapy for children with severe bone disease
Part of Bones, joints & muscles clinical trials.
This trial tests a gene therapy that uses a child's own stem cells to treat autosomal recessive osteopetrosis (a genetic bone disease). It aims to improve bone health and reduce complications like fractures and vision loss.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be between 28 days and 2 years old and weigh at least 9 pounds.
- Have a diagnosis of autosomal recessive osteopetrosis caused by a specific gene change (TCIRG1).
- Must have adequate heart, lung, kidney, and liver function.
- Cannot have a fully matched sibling or unrelated donor available for a standard stem cell transplant.
- Must not have an active infection, cancer, or a history of seizures.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a one-time gene therapy using your own stem cells to help you produce healthy red blood cells and reduce or eliminate the need for regular blood transfusions. It is for people with transfusion-dependent beta-thalassemia who are between 3 and 35 years old.
This trial tests a gene therapy called ECUR-506 for baby boys under 9 months old with a severe form of OTC deficiency. The goal is to see if it can help their bodies process ammonia better, reducing the need for a strict protein-restricted diet and medications.
This trial tests a one-time gene replacement therapy given into the fluid around the brain to help children with CTNNB1 neurodevelopmental syndrome. The goal is to see if replacing the faulty gene can improve development and reduce symptoms.
This trial tests a one-time gene therapy delivered into the ear to improve hearing in people with certain OTOF gene changes. It studies safety and how well the treatment can work, especially in children and infants, as doctors monitor hearing tests for up to 48 weeks.
This early-phase study tests an AAV gene therapy meant to reduce ammonia levels in children with ornithine transcarbamylase (OTC) deficiency. It’s designed to slow or stop ammonia-related harm, while also carefully monitoring safety for years.
This trial tests a cell-based treatment to improve bone strength in children with the most severe type of osteogenesis imperfecta (brittle bone disease). If you qualify, it may help slow bone weakening and support growth.
Hear when a new Osteopetrosis trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.